The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.
FDA inspectors have cited the manufacturer of the rare-disease drug Soliris (eculizumab) for poor manufacturing practices.
The oral medication Cerdelga joins Cerezyme in its Gaucher disease arsenal. Sanofi expects the drug will be priced "on par" with Cerezyme, which goes for around $300,000 a year in the US.
Phase-III tests indicate patients may be able to switch from injectable enzyme-replacement therapies, like Fabrazyme, to the firm's oral drug.
A $225-million deal with US biotech ArmaGen gives Shire worldwide commercialization rights to an experimental enzyme replacement therapy.
An advocacy group alleges that Shire's decision to ignore the US Fabry disease market was the result of a backroom deal with Sanofi.
Analyst reaction to this week's quarterly news reflected a need to woo investors, adding to some negative carryover from the previous year.
The company said it plans to file its rare-disease drug with the FDA later this year, for a 2015 review.
The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.
The UK watchdog wants to know what's behind the rare-disease drug's high price.
Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.
Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.
The FDA granted priority review for the experimental, all-oral Gaucher's disease treatment.
The agency extended the review time for Biogen's Alprolix by three months.
The patient population is small, at around 3,000, but the expected price tag is $330,000 a year.
The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.
The US-only recall amounts to less than 1% of the company's inventory.
The regulator says the drugmaker needs to do more testing.
Eli Lilly is the first Big Pharma to team up with the agency in its rare-disease pre-clinical development program.
Price, market potential and emotional pull make this subcategory of orphan diseases one to watch, an analyst writes. Hint: it's not oncology.
Takeda moves an Alzheimer's treatment to Phase III; Sangamo acquires Ceregene; Catalyst gets breakthrough designation for auto-immune disorder drug; study says seniors likely to opt out of health insurance
Australia's price watchdogs are assessing the value of BMS cancer drug Yervoy; why a Phase II weight drug from Zafgen could open up the category; GSK halts a Crohn's trial; and Shire extends Santaris rare-disease collaboration
The drug maker scored its third breakthrough designation label, this time for orphan drug bimagrumab, a possible treatment for a muscle-wasting disease.
Alexion plans on moving into new HQ in 2015, AMA declares obesity is a disease, FDA looks into Zyprexa patient deaths
Genzyme is taking rare disease awareness to another level with a documentary film, dubbed "Facing Fabry Together," that follows four families grappling with the genetic disorder.
A study shows FDA approvals and awareness efforts are having little impact on physician awareness or patient frustration.
Industry observances of Rare Disease Awareness Day are getting more elaborate of late—perhaps not solely for altruistic reasons, as a third of NMEs approved by FDA over the past five years were for the treatment of rare diseases.
Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.
A recent study says that the financial incentives go beyond taxes and grants and that investing in R&D can mean entry into a space that's grown 26% from 2001 to 2010.
PR firm Burson-Marsteller launched a new healthcare subsidiary, Orexigen's net loss widened in Q2, and Corcept closed out its first full quarter with orphan treatment Korlym.
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